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斑马鱼在遗传性视网膜变性疾病建模与治疗发现中的作用

The Power of Zebrafish in Disease Modeling and Therapy Discovery for Inherited Retinal Degeneration.

作者信息

Xiao Huanhuan, Marshall Randi, Saxena Meera T, Zhang Liyun

机构信息

Department of Ophthalmology, Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD, USA.

出版信息

Adv Exp Med Biol. 2025;1468:229-233. doi: 10.1007/978-3-031-76550-6_38.

DOI:10.1007/978-3-031-76550-6_38
PMID:39930201
Abstract

In the research of inherited retinal degeneration (IRD), zebrafish have emerged as a powerful model system, offering profound insights into disease mechanisms and opening new therapeutic avenues. This mini-review discusses the distinctive advantages that zebrafish provide for investigating retinal degeneration. It outlines contemporary genetic tools, with a specific focus on advanced CRISPR/Cas9 gene targeting technology, utilized for genome manipulation and disease modeling in zebrafish. By emphasizing the pivotal role of zebrafish in large-scale high-throughput drug discovery and the exploration of innovative gene therapy strategies, this succinct review underscores the adaptability and significance of the zebrafish model in advancing IRD research. It establishes a robust foundation for future studies and therapeutic developments in the field.

摘要

在遗传性视网膜变性(IRD)的研究中,斑马鱼已成为一个强大的模型系统,为疾病机制提供了深刻见解,并开辟了新的治疗途径。这篇综述讨论了斑马鱼在研究视网膜变性方面所具有的独特优势。它概述了当代遗传工具,特别关注用于斑马鱼基因组操作和疾病建模的先进CRISPR/Cas9基因靶向技术。通过强调斑马鱼在大规模高通量药物发现和创新基因治疗策略探索中的关键作用,这篇简洁的综述强调了斑马鱼模型在推进IRD研究中的适应性和重要性。它为该领域未来的研究和治疗发展奠定了坚实基础。

相似文献

1
The Power of Zebrafish in Disease Modeling and Therapy Discovery for Inherited Retinal Degeneration.斑马鱼在遗传性视网膜变性疾病建模与治疗发现中的作用
Adv Exp Med Biol. 2025;1468:229-233. doi: 10.1007/978-3-031-76550-6_38.
2
CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration.CRISPR-Cas9 基因组工程:治疗遗传性视网膜变性。
Prog Retin Eye Res. 2018 Jul;65:28-49. doi: 10.1016/j.preteyeres.2018.03.003. Epub 2018 Mar 22.
3
Gene Therapy for Inherited Retinal Degeneration.遗传性视网膜变性的基因治疗。
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Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.诱导多能干细胞和基因组编辑工具在确定遗传性视网膜疾病的基因功能和治疗中的应用。
Int J Mol Sci. 2022 Dec 3;23(23):15276. doi: 10.3390/ijms232315276.
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Application of CRISPR/Cas9 technologies combined with iPSCs in the study and treatment of retinal degenerative diseases.CRISPR/Cas9 技术与 iPSCs 的联合应用在视网膜退行性疾病的研究和治疗中的应用。
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Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration.使用 CRISPR-Cas9 生成基因矫正的自体诱导多能干细胞治疗遗传性视网膜变性。
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Invest Ophthalmol Vis Sci. 2020 Apr 9;61(4):31. doi: 10.1167/iovs.61.4.31.

本文引用的文献

1
A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for -associated retinitis pigmentosa.一种面向蛋白质结构域的方法,以扩大与相关视网膜色素变性的治疗性外显子跳跃的机会。
Mol Ther Nucleic Acids. 2023 May 20;32:980-994. doi: 10.1016/j.omtn.2023.05.020. eCollection 2023 Jun 13.
2
Retinal organoids provide unique insights into molecular signatures of inherited retinal disease throughout retinogenesis.视网膜类器官在整个视网膜发生过程中提供了对遗传性视网膜疾病分子特征的独特见解。
J Anat. 2023 Aug;243(2):186-203. doi: 10.1111/joa.13768. Epub 2022 Sep 29.
3
Zebrafish and inherited photoreceptor disease: Models and insights.
斑马鱼与遗传性光感受器疾病:模型与研究进展。
Prog Retin Eye Res. 2022 Nov;91:101096. doi: 10.1016/j.preteyeres.2022.101096. Epub 2022 Jul 8.
4
Large-scale phenotypic drug screen identifies neuroprotectants in zebrafish and mouse models of retinitis pigmentosa.大规模表型药物筛选鉴定出斑马鱼和视网膜色素变性小鼠模型中的神经保护剂。
Elife. 2021 Jun 29;10:e57245. doi: 10.7554/eLife.57245.
5
Drug screening with zebrafish visual behavior identifies carvedilol as a potential treatment for an autosomal dominant form of retinitis pigmentosa.利用斑马鱼视觉行为进行药物筛选,鉴定卡维地洛可作为治疗常染色体显性遗传型视网膜色素变性的一种潜在疗法。
Sci Rep. 2021 Jun 1;11(1):11432. doi: 10.1038/s41598-021-89482-z.
6
A Zebrafish Model of Retinitis Pigmentosa Shows Continuous Degeneration and Regeneration of Rod Photoreceptors.《斑马鱼视网膜色素变性模型中视杆细胞的持续变性与再生》
Cells. 2020 Oct 6;9(10):2242. doi: 10.3390/cells9102242.
7
Fovea-like Photoreceptor Specializations Underlie Single UV Cone Driven Prey-Capture Behavior in Zebrafish.类黄斑光感受器特化结构是斑马鱼单根紫外光视锥细胞驱动捕食行为的基础。
Neuron. 2020 Jul 22;107(2):320-337.e6. doi: 10.1016/j.neuron.2020.04.021. Epub 2020 May 29.
8
Understanding the genetic architecture of human retinal degenerations.了解人类视网膜变性的遗传结构。
Proc Natl Acad Sci U S A. 2020 Feb 25;117(8):3904-3906. doi: 10.1073/pnas.1922925117. Epub 2020 Feb 7.
9
Highly Efficient CRISPR-Cas9-Based Methods for Generating Deletion Mutations and F0 Embryos that Lack Gene Function in Zebrafish.高效的基于 CRISPR-Cas9 的方法用于生成缺失突变和缺乏基因功能的 F0 胚胎在斑马鱼中。
Dev Cell. 2019 Dec 2;51(5):645-657.e4. doi: 10.1016/j.devcel.2019.10.004. Epub 2019 Nov 7.
10
ARAF recurrent mutation causes central conducting lymphatic anomaly treatable with a MEK inhibitor.ARAF 反复突变导致中央传导性淋巴异常,可用 MEK 抑制剂治疗。
Nat Med. 2019 Jul;25(7):1116-1122. doi: 10.1038/s41591-019-0479-2. Epub 2019 Jul 1.